The new investigation left good results for those known as “butterfly children”. And that’s what experts discovered about the first gene therapy on it Skin Heal these people Wounds Which has not been cured for years. This therapy is applied directly to the skin and Cured nine patients with this diseaseKnown as Bullous epidermolysis, Any contact causes terrible injuries.
In fact, the specialists of these “butterfly children” point out that there are wounds that can last for years openly and without healing. In this sense, epidermolysis bullosa is a combination of genetic disorders that cause skin hypersensitivity, producing Fragile skin with blisters.
People known as “butterfly children” get this name because the disease begins to develop from birth and makes the skin as soft as the wings of this insect.
People with this disease have a defect in the COL7A1 gene, which means that people can not synthesize the protein collagen 7. This protein is responsible for retaining the two outermost layers of the skin: the epidermis and the dermis.
People with epidermolytic bullosa cause open sores and wounds all over the body with a slight touch.
Encouraging results for “Butterfly for children”
The latest study of this disease has left a good taste in the mouth. And that is that nine patients with this disease underwent new therapy. The study was conducted at Stanford University (USA) and was led by Stanford University dermatologist Peter Marinkovich.
The work, published in Nature Medicine, is based on the modified herpes virus, which contains two healthy copies of the COL7A1 gene. So, as experts explain, when an inactivated pathogen enters the cells, they begin to produce a protein that corrects the disease.
As specialists point out, the most striking thing about this therapy is that it is applied directly to the skin in drops, without touching the lesions. And the results were a complete success. Most of the wounds healed after treatment and remained closed for at least three months. In contrast, wounds that had not been treated healed much less.
“This therapy strengthens the skin and breaks the cycle of opening and healing of chronic wounds in patients,” explained Peter Marinkovich.
New Gene Therapy News
Unlike the therapies that have always been used during this disease, this new treatment does not require hospitalization. Earlier methods of curing this disease were based on autotransplantation.
In this case, a sample of the patient’s skin was taken and his cells were processed. All this to correct a genetic defect. The tissue was then cultured until it had almost a square foot of healthy skin to be transplanted with personalized and complex intervention on the patient.
In turn, new gene therapy can be administered periodically without serious side effects. However, it does not permanently correct a genetic defect.
The key to this new treatment is that the patient’s cells produced the long-awaited collagen seven on the day of taking the drug, which allows the wounds to close.
There is still a long way to go as the project is in its final phase but the results are very positive. “These results have very important implications because they can transform the field of gene therapy,” experts say.
Source: El Diario
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